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Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations

This study is currently recruiting participants.
See Contacts and Locations
Verified December 2016 by Spark Therapeutics
Sponsor:
Collaborators:
Children's Hospital of Philadelphia
University of Pennsylvania
Massachusetts Eye and Ear Infirmary
Information provided by (Responsible Party):
Spark Therapeutics
ClinicalTrials.gov Identifier:
NCT02341807
First received: January 12, 2015
Last updated: December 20, 2016
Last verified: December 2016
  Purpose
This clinical study evaluates the safety and tolerability of AAV2-hCHM in subjects with Choroideremia gene mutations. Two dose groups will be evaluated.

Condition Intervention Phase
Choroideremia CHM (Choroideremia) Gene Mutations Biological: AAV2-hCHM Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the Retina

Resource links provided by NLM:


Further study details as provided by Spark Therapeutics:

Primary Outcome Measures:
  • Safety and tolerability (assessed by physical exam, vital signs, laboratory changes over time, and adverse events) [ Time Frame: 2 years ]
    Safety and tolerability of a single dose of AAV2-hCHM will be assessed by physical exam, vital signs, laboratory changes over time, and adverse events.


Estimated Enrollment: 15
Study Start Date: January 2015
Estimated Study Completion Date: January 2019
Estimated Primary Completion Date: January 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dose Group 1
Single, unilateral administration of a single low dose range of AAV2-hCHM.
Biological: AAV2-hCHM
Comparison of different dosages of AAV2-hCHM
Experimental: Dose Group 2
Single, unilateral administration of a single high dose range of AAV2-hCHM.
Biological: AAV2-hCHM
Comparison of different dosages of AAV2-hCHM

Detailed Description:
The primary objective is to evaluate the safety and tolerability of subretinal administration of AAV2-hCHM, in an inter-subject group dose escalation in individuals with choroideremia, based on a comprehensive clinical monitoring plan. The secondary objectives are to define the dose of AAV2-hCHM required to achieve stable, or improved, visual function/functional vision and to assess development of immune responses to AAV2 and REP-1.
  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male at least 18 years of age diagnosed with CHM gene mutation
  • Central visual field (VF) < 30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected
  • Any evidence of functioning outer retinal cells within the central 10°

Exclusion Criteria:

  • Previous history of ocular inflammatory disease (uveitis)
  • Prior intraocular surgery within six months
  • Participation in a previous gene therapy research trial within one year of enrollment or participation in any other ocular gene therapy trial
  • Participation in a clinical study with an investigational drug in the past six months
  • Grossly asymmetrical disease, or other eye morbidity, which may render the contralateral eye ineffective as a control
  • Visual acuity < 20/200 on standard ETDRS testing in the eye to be injected
  • Presence of disease which may preclude the subject from participation in this trial
  • Use of medications known to be neuroprotective or retino-toxic that could potentially interfere with the disease process and/or cause ocular adverse events; individuals who discontinue use of these compounds for 6 months may become eligible
  • Identification by the investigator as being unable or unwilling to perform / be compliant with study procedures.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02341807

Contacts
Contact: Spark Therapeutics clinicaltrials@sparktx.com

Locations
United States, Massachusetts
Massachusetts Eye and Ear Infirmary Recruiting
Boston, Massachusetts, United States, 02114
United States, Pennsylvania
University of Pennsylvania Active, not recruiting
Philadelphia, Pennsylvania, United States, 19014
Children's Hospital of Philadelphia Active, not recruiting
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Spark Therapeutics
Children's Hospital of Philadelphia
University of Pennsylvania
Massachusetts Eye and Ear Infirmary
Investigators
Study Director: Clinical Director Spark Therapeutics
  More Information

Responsible Party: Spark Therapeutics
ClinicalTrials.gov Identifier: NCT02341807     History of Changes
Other Study ID Numbers: AAV2-hCHM-101
Study First Received: January 12, 2015
Last Updated: December 20, 2016

Keywords provided by Spark Therapeutics:
Choroideremia
AAV
Gene therapy
CHM
Adeno-associated virus
Adeno-associated viral vector

Additional relevant MeSH terms:
Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on June 23, 2017